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A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects | Clinical Trials | Clareo Health

COMPLETEDPHASE1

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular Dystrophy

NCT01128855•GlaxoSmithKline

View on ClinicalTrials.gov

Summary

The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy

Eligibility

Age

9 - No limit years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK2402968.
•Age 9 years old or greater at Screening;
•Male;
•Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
•Life expectancy at least three years;
•Willingness and ability to comply with all protocol requirements and procedures;
•QTc \<450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread;
•Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug;
•Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).

Exclusion Criteria

•Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968;
•Current or history of liver or renal disease;
•Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments;
•Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication;
•Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication;
•Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening;
•Symptomatic cardiomyopathy;
•Use of alcohol from Screening through to the 1 month Follow-up visit ;
•Any Child in Care.

Locations (2)

GSK Investigational Site

Columbus, Ohio, United States

GSK Investigational Site

Paris, France

Key Dates

Start Date

July 12, 2010

Primary Completion Date

October 25, 2011

Completion Date

October 25, 2011

Last Updated

July 18, 2017

Enrollment

ACTUAL participants

Conditions

Muscular Dystrophies

Interventions

3 mg/kg GSK2402968

DRUG

6 mg/kg GSK2402968

DRUG

9 mg/kg GSK2402968

DRUG

12 mg/kg GSK2402968

DRUG

Placebo

OTHER

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 18, 2017Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

Inclusion Criteria

Exclusion Criteria

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